A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)

Study type

Interventional

Primary purpose

Treatment

Allocation

Randomized

Intervention model

Parallel

Masking

None

Arms

3

Interventions

• Deflazacort Drug

• Duchenne Muscular Dystrophy

Primary outcome

Period 1 and 2: Number of Participants With Treatment Emergent Adverse Events (TEAEs) (52 weeks); Period 1 and 2: Change From Baseline in Vital Signs and Electrocardiogram (ECG) at Week 52 (Baseline, Week 52); Period 1 and 2: Change From Baseline in the Child Behavior Checklist Score at Week 52 (Baseline, Week 52); Period 1 and 2: Change From Baseline in the Normalized Measure of Bone Density Change (Z-score) for the Dual Energy X-ray Absorptiometry (DEXA) at Week 52 (Baseline, Week 52); Period 1 and 2: Mean Change From Baseline in Height at Week 52 (Baseline, Week 52); Period 1 and 2: Mean Change From Baseline in Body Weight at Week 52 (Baseline, Week 52); Period 1 and 2: Mean Change From Baseline in Height Percentile for Age at Week 52 (Baseline, Week 52); Period 1 and 2: Number of Participants With Clinically Significant Laboratory Tests (52 weeks)

• Deflazacort

• NCT03642145 on ClinicalTrials.gov (opens in a new tab) Licence: public-domain. Read 25 Oct 2026

Source: ClinicalTrials.gov, U.S. National Library of Medicine. Listing a study does not mean NLM has evaluated it.